PharmaMar

Aplidin(R) Granted Orphan Drug Status by the European Commission for the Treatment of Multiple Myeloma

Madrid, November 19 (ots/PRNewswire) - PharmaMar announced today that Aplidin(R) (1), its second marine-derived compound in clinical development, was granted orphan drug status by the European Commission for the treatment of multiple myeloma (MM) following adoption of a positive opinion by the Committee for Orphan Medicinal Products (COMP) of the European Agency for the Evaluation of Medicinal Products (EMEA). Last September, the Food & Drug Administration (FDA) granted orphan drug status to Aplidin for the same indication in the United States. In July 2003, Aplidin(R) was granted orphan drug status in the European Union for the treatment of acute lymphoblastic leukaemia (ALL), a malignant disease of the bone marrow. Orphan drug designation in the European Union offers several incentives, such as 10-years marketing exclusivity throughout Europe for the therapeutic indication for which it was granted, protocol assistance from the EMEA to optimise drug development, reduction of fees for all types of centralised procedure activities and access to grants from EC and Member States programmes. Multiple myeloma is the second most common haematological malignancy after Non-Hodgkin's Lymphoma and is part of a spectrum of diseases labelled Plasma Cell Dyscrasia. Plasma cells are the cells responsible for forming antibodies against bacteria and foreign agents. For reasons that are unclear, these cells lose their ability to respond to controlling signals from a hierarchy of immune cells. Plasma cells then divide with no control and form abnormal proteins, which results in damage to the bone, the kidney, and/or other organs of the body. Incidence and mortality rates are similar in Europe and in the United States, being close to 5/100,000 and 4/100,000 inhabitants respectively. It accounts for about 1% of all cancer types and for 10% of all hematological malignancies. The survival rate for MM has not changed for the past three decades, with only about 28% of patients surviving for longer than five years. No single standard therapy currently exists for multiple myeloma patients that have relapsed or become resistant to treatment and these patients have an expected survival of just 6-9 months. Despite high-dose chemotherapy treatments and the appearance of some new agents with significant activity in the relapsed disease, MM remains an incurable disease, with 10-year survival rates estimated to be less than 5% (Medscape CME 2003). Isabel Lozano, Chief Executive Officer of PharmaMar, said: "This orphan drug designation in Europe recognises the urgent need for new therapies for the treatment of multiple myeloma. This will support the sustained clinical development of Aplidin in MM, as well as in other haematological malignancies, which will start before the year end." About Aplidin(R) Aplidin is a cyclic peptide, originally isolated from the marine tunicate Aplidium albicans, currently obtained by total synthesis. It induces apoptosis rapidly and persistently, inhibits VEGF secretion and blocks cell-cycle. It is currently in therapeutic exploratory clinical evaluation (phase II) in solid and haematological malignancies, including paediatrics. The clinical program involves hospitals in Europe, Canada and the US. Approximately 400 patients have been treated. In the preclinical development, human leukaemia, myeloma and lymphoma tumour cell lines are particularly sensitive to Aplidin. There is no evidence of cross-resistance with commonly used therapeutic agents for haematological malignancies. There is no clinical evidence of relevant bone marrow toxicity. Its side effects are reversible and manageable (including muscular and liver biochemical alterations). Hair loss and oral ulcers are infrequent. Aplidin was granted Orphan Drug Designations for the treatment of Acute Lymphoblastic Leukaemia in the European Union in 2003 and in the US in 2004.The US FDA also granted ODD for Multiple Myeloma in 2004. (1) Aplidin(r) is a PharmaMar registered trademark. PharmaMar PharmaMar is a biopharmaceutical company, advancing cancer care through the discovery and development of innovative marine-derived medicines. PharmaMar's clinical portfolio currently includes Yondelis (TM) in phase II clinical trials (co-developed with Johnson & Johnson Pharmaceutical Research & Development), designated Orphan Drug for STS by the European Commission (E.C.) in 2001 and by the FDA in 2004, and Orphan Drug for ovarian cancer by the E.C. in 2003; Aplidin(R), in phase II, designated Orphan Drug for acute lymphoblastic leukaemia by the E.C. in 2003 and by the FDA in 2004, and for multiple myeloma by the FDA in 2004; Kahalalide F in phase II and ES-285 in phase I clinical trials. PharmaMar, based in Madrid, Spain, is a subsidiary of the Zeltia Group (Spanish stock exchange: ZEL.MC; Bloomberg: ZEL SM; Reuters: ZEL.MC). PharmaMar can be found on the Web at http://www.pharmamar.com ots Originaltext: PharmaMar Im Internet recherchierbar: http://www.newsaktuell.ch Contact: Lola Casals, PharmaMar, Tel: +34-91-846-6000. David Yates & Deborah Scott, Financial Dynamics, Tel: +44-207-831-3113

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